Trial results for the BLAST MRD AML-2 study (NCT04284787) investigating pembrolizumab in combination with azacitidine and venetoclax for acute myeloid leukemia were posted on ClinicalTrials.gov on 2025-03-13. The Phase 2 study indicated that adding pembrolizumab did not increase the percentage of patients achieving minimal residual disease-negative complete remission compared to azacitidine and venetoclax alone.

Background

The BLAST MRD AML-2 study (NCT04284787) investigated the addition of pembrolizumab to standard therapy with azacitidine and venetoclax for newly diagnosed, unfit patients with acute myeloid leukemia. The trial specifically targeted measurable residual disease (MRD) in this patient population.

Trial design

The BLAST MRD AML-2 study (NCT04284787) was a Phase 2, randomized study that enrolled 60 participants. The trial investigated pembrolizumab in combination with azacitidine and venetoclax as frontline therapy for unfit patients with acute myeloid leukemia, including those with acute myeloid leukemia arising from previous myelodysplastic syndrome, post cytotoxic therapy, or secondary acute myeloid leukemia. Participants were assigned to either Arm I, receiving azacitidine and venetoclax, or Arm II, receiving azacitidine, venetoclax, and pembrolizumab. The study assessed the percentage of patients achieving minimal residual disease-negative complete remission (MRD-CR) or MRD-complete remission with incomplete count recovery (CRi).

Key results

The trial results for the outcome "Percentage of Patients With Minimal Residual Disease Negative Complete Remission (MRD-CR) or MRD-complete Remission With Incomplete Count Recovery (Cri) With Azacitidine (AZA) + Venetoclax (VEN) With MK-3475 (Pembrolizumab)" showed identical participant counts across both treatment arms for different time points or sub-analyses (the data does not specify the different instances of the same outcome title). For Arm I (AZA, VEN), the counts were 13, 7, and 9 participants. For Arm II (AZA, VEN, Pembrolizumab), the counts were also 13, 7, and 9 participants for the same respective measurements. Additionally, for the outcome "Proportion of Patients Who Develop Severe Toxicity" in Arm II (AZA, VEN, Pembrolizumab), 0 participants developed severe toxicity.

What this means

The identical participant counts for minimal residual disease-negative complete remission (MRD-CR) or MRD-complete remission with incomplete count recovery (CRi) across both the azacitidine + venetoclax arm and the azacitidine + venetoclax + pembrolizumab arm suggest that the addition of pembrolizumab did not enhance this outcome in the studied population of unfit acute myeloid leukemia patients. The finding of 0 participants developing severe toxicity in the pembrolizumab combination arm suggests a potentially tolerable safety profile, though comprehensive safety data would be needed for a full assessment.

Source

The information regarding these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT04284787, titled "BLAST MRD AML-2: BLockade of PD-1 Added to Standard Therapy to Target Measurable Residual Disease in Acute Myeloid Leukemia 2- A Randomized Phase 2 Study of Anti-PD-1 Pembrolizumab in Combination With Azacitidine and Venetoclax as Frontline Therapy in Unfit Patients With Acute Myeloid Leukemia," were posted on 2025-03-13 on clinicaltrials.gov.